A landmark gene-editing treatment for sickle cell disease moves closer to reality
Advisers to the Food and Drug Administration meet Tuesday to review the first treatment of human disease using the gene-editing technique CRISPR. The agency has a December deadline to make a decision.
The Food and Drug Administration may soon approve a therapy that uses the gene-editing technique called CRISPR to treat sickle cell disease. It would mark the first time gene editing moves from the lab into clinical use.
A committee of advisers to the FDA meets Tuesday to review the scientific evidence for the treatment, including whether sufficient research has been done to assess its long-term safety. The agency has until Dec. 8 to make a decision about the therapy, known as "exa-cel."
In an Friday, agency scientists conclude exa-cel is highly effective at preventing episodes of excruciating pain that plague sickle cell disease patients. The treatment worked
